Spastic Paraplegia, Hereditary

Reported Full Year 2023 total revenues of 2% growth at €48.5 million Secured approximately USD 20 million – strengthening cash position and expanding relationship with Lifera, a biopharma company owned by the PIF Positioned for strong performance in 2024, driven by new and existing Pharma business and solid Diagnostic growth in line with industry standards; guidance of FY2024 total revenue growth between 10-15% Ongoing strategic alternatives process focused on sustainable long-term value creation for the benefit of its stakeholders CAMBRIDGE, Mass. and ROSTOCK, Germany and BERLIN, May 15, 2024 (GLOBE NEWSWIRE) -- Centogene N.V. (Nasdaq: CNTG) (“we” or the “Company”), the essential life science partner for data-driven answers in rare and neurodegenerative diseases, today announced financial results for the fiscal year ended December 31, 2023, and provided a business update. “In 2023, we focused on strategic growth – strengthening our Diagnostic portfolio and introducing new data and wet lab solutions to accelerate drug discovery, development, and commercialization for Pharma. We expanded our partnership model with a joint venture in Saudi Arabia, securing a $30 million investment and additional milestone payments. However, delays in Pharma program timelines impacted our 2023 revenue. Looking ahead to 2024, our Diagnostics business is expected to maintain steady growth in line with industry standards, and our Pharma orderbook is on a record trajectory – with momentum expected to compound each quarter.” Kim Stratton continued, “We are excited to be engaged in discussions with multiple potential strategic partners and to have deepened our relationship with Lifera over the past months. With this progress at the forefront, CENTOGENE is positioned for growth and a path towards profitability. We expect to see 2024 annual revenue growth between 10-15%.” FullYear2023FinancialHighlights Total revenues increased by 2% to €48.5 million in FY2023, compared to €47.5 million in FY2022 Diagnostics segment revenues increased by 8% in 2023 to €33.7 million, primarily related to an increase of 11% in revenues for CentoXome® (CENTOGENE’s proprietary Whole Exome Sequencing) and CentoGenome® (CENTOGENE’s proprietary Whole Genome Sequencing). Achieved upselling 47% of CentoXome® and CentoGenome® orders to MOx (CENTOGENE’s portfolio of multiomic testing solutions) in FY2023 Pharma segment revenues of €14.8 million, reflecting a decrease of 8%, primarily driven by a lag in Pharma program timelines in H2 2023 Overall gross profit margin of 36% of revenues or €17.2 million in FY2023, compared to 42% of revenues or €19.8 million in FY2022 Net loss position increased by 12.2% at €35.8 million in FY2023 compared to a net loss of €31.9 million in FY2022 Total segment adjusted EBITDA was at €11.1 million in FY2023, compared to €13.2 million in FY2022. This decrease was mainly driven by the lag in pharma revenues, as well as the increase in cost of sales and selling expenses Cash and cash equivalents were €19.1 million as of December 31, 2023, compared to €35.9 million for the period ended December 31, 2022 “We are focused on streamlining processes and strengthening our financial position to enable us to execute on our leading rare and neurodegenerative disease business and sustainable long-term value creation,” said Miguel Coego, Chief Financial Officer at CENTOGENE. “Looking ahead, we will remain prudent with our capital allocation – serving as a key driver to our goal of hitting EBITDA breakeven by the end of this year and our path to cash profitability. We believe this will enable us to advance our strategic alternatives process to unlock value for our patients, physicians, pharma partners, and CENTOGENE stakeholders.” RecentBusiness Highlights Corporate Announced strategic collaboration with Lifera, a biopharmaceutical company wholly-owned by the PIF, with the formation of a joint venture (JV), Lifera Omics, to increase local and regional access and rapid delivery of world-class genomic and multiomic testing to patients in Saudi Arabia and countries of the Gulf Cooperation Council (GCC). Under the terms of the collaboration, CENTOGENE received a $30 million mandatory convertible loan from Lifera, as well as milestone payments Secured an additional approximately $20 million proceeds from Lifera via the sale of $15 million in CENTOGENE’s accounts receivables (AR) in Saudi Arabia at face value with no recourse. Additionally, Lifera purchased 16% of CENTOGENE’s stake into their JV at a value of approximately $5 million with CENTOGENE maintaining 4% ownership in the JV with the option to repurchase the previous ownership on substantially the same terms in the next 6-24 months. CENTOGENE to remain active in Lifera Omics’ operations to deliver genomic and multiomic testing Initiated strategic alternatives process focused on sustainable long-term value creation for the benefit of its stakeholders Expanded the CENTOGENE Biodatabank to over 850,000 patients, over 70% of whom are of non- European descent, approximately 30,000 active physicians, and more than 85 million unique variants thanks to the increasing number of CentoXome® and CentoGenome® analyses, which contain significantly more variants than more targeted diagnostic tests Authored over 30 peer-reviewed scientific publications in FY2023, reaching a milestone of over 300 publications in the Company’s history. The research unlocked insights into Parkinson’s disease, Gaucher disease, Niemann-Pick type C1 disease, hereditary angioedema, autosomal recessive spastic paraplegia, colorectal carcinomas, renal hypouricemia, CLN6 disease, ELOVL4-related autosomal recessive neuro-ichthyosis, CFTR-related disease, TOR1A-related disorders, as well as a range of variants associated with epilepsy, genetic cancers, metabolic disorders, developmental disorders, and other rare and neurodegenerative diseases Pharma Led 48 collaborations with 34 different pharmaceutical partners in FY2023. Of these 48 collaborations, 12 were with new partners. Starting off Q1 2024, the Company has formed collaborations with eight new partners Extended Takeda partnership to March 2026 to continue providing access to genetic testing for patients with lysosomal storage disorders Announced research project with The Michael J. Fox Foundation to validate the genetic risk factors of Parkinson’s disease using multiomics Reached initial recruitment and genetic testing milestone in the observational EFRONT Study, being conducted to advance the genetic understanding of frontotemporal dementia (FTD) Leading, alongside Denali Therapeutics, the ROPAD Study, the world’s largest observational study on Parkinson’s disease genetics with over 15,000 enrolled patients to date. Patients enrolled in ROPAD and identified with LRRK2 genetic variations may be eligible for participation in ongoing interventional clinical studies Diagnostics Strong test requests of approximately 81,500 test requests for FY2023, representing an increase of approximately 18% as compared to approximately 57,100 in the prior year Strengthened Diagnostics Sales team and expanded direct footprint distribution network in targeted geographic areas, such as Canada, Colombia, Italy, Spain, and Portugal Expanded MOx, the Company’s multiomic diagnostic portfolio, now incorporating cutting-edge transcriptomic analysis. CENTOGENE’s MOx 2.0 is a single-step multiomic solution that combines DNA sequencing, biochemical testing, and now RNA sequencing to provide physicians with the most comprehensive testing capability Launched NEW CentoGenome®, the world's most comprehensive Whole Genome Sequencing tool for diagnosis of rare and neurodegenerative diseases, which now detects Copy Number Variations associated with spinal muscular atrophy, as well as complex disease-causing variants associated with Gaucher disease and susceptibility to GBA1-related Parkinson's disease Launched CENTOGENE’s FilterTool to fuel rapid, reliable analysis for diagnosis and research of rare genetic diseases by significantly reducing time- and resource-intensive processes – enabling users to display, filter, select, and classify variants. Seamlessly integrating with CentoCloud®, CENTOGENE’s CE-marked Software as a Service (SaaS) bioinformatics pipeline, FilterTool is one of the first applications to receive CE mark under new In Vitro Diagnostic Regulation (IVDR) from the European Parliament Launched together with TWIST Bioscience three Next Generation Sequencing target enrichment panels, Twist Alliance CNTG Exome, Twist Alliance CNTG Rare Disease Panel, and Twist Alliance CNTG Hereditary Oncology Panel, to support rare disease and hereditary cancer research and support diagnostics Integrated Illumina’s new NovaSeq X Plus Sequencer into the Company’s state-of-the-art, CAP/CLIA accredited laboratory in Rostock, Germany, to further optimize throughput, scale, and cost efficiencies Published research in Science in collaboration with the Laboratory of Human Genetics of Infectious Diseases at Institut Imagine on human pre-T cell receptor alpha (pre-TCRα) deficiency and its effect on human immunity Published study in the European Journal of Human Genetics revealing unique genetic variants in world's largest Niemann-Pick type C1 disease cohort Published the discovery of a new form of early-onset dystonia and parkinsonism in the context of neurodevelopmental abnormalities associated to the gene called ACBD6 (Acyl-CoA Binding Domain Containing 6) as part of an international team of researchers. The landmark study’s findings have been published in Brain Published a study in the Diagnostics journal establishing lyso-Gb1 (glucosylsphingosine) as a predictive biomarker 2024 Revenue Guidance The Company expects revenue growth to be between 10-15% in FY2024 compared to FY2023. Update on Process to Review Strategic Alternatives On February 28, 2024, we announced a process to explore strategic alternatives, including the sale of the Company, divestitures of asset, licensing/partnership transactions, and/or additional financing. We engaged an investment banker firm to advise us in connection with this process. We are currently in active discussions with several interested parties, which could result in a near-term transaction by July 15, 2024. We do not expect to disclose developments unless and until our board of directors has concluded that disclosure is appropriate or required. There can be no assurance that our strategic review process will result in any transaction or other strategic outcome. We do not intend to disclose further developments on this strategic review process unless and until we determine that such disclosure is appropriate or necessary. AboutCENTOGENE CENTOGENE’s mission is to provide data-driven, life-changing answers to patients, physicians, and pharma companies for rare and neurodegenerative diseases. We integrate multiomic technologies with the CENTOGENE Biodatabank – providing dimensional analysis to guide the next generation of precision medicine. Our unique approach enables rapid and reliable diagnosis for patients, supports a more precise physician understanding of disease states, and accelerates and de-risks targeted pharma drug discovery, development, and commercialization. Since our founding in 2006, CENTOGENE has been offering rapid and reliable diagnosis – building a network of approximately 30,000 active physicians. Our ISO, CAP, and CLIA certified multiomic reference laboratories in Germany utilize Phenomic, Genomic, Transcriptomic, Epigenomic, Proteomic, and Metabolomic datasets. This data is captured in our CENTOGENE Biodatabank, with over 850,000 patients represented from over 120 highly diverse countries, over 70% of whom are of non-European descent. To date, the CENTOGENE Biodatabank has contributed to generating novel insights for more than 300 peer-reviewed publications. By translating our data and expertise into tangible insights, we have supported over 50 collaborations with pharma partners. Together, we accelerate and de-risk drug discovery, development, and commercialization in target and drug screening, clinical development, market access and expansion, as well as offering CENTOGENE Biodata Licenses and Insight Reports to enable a world healed of all rare and neurodegenerative diseases. To discover more about our products, pipeline, and patient-driven purpose, visit and follow us on LinkedIn. Forward-Looking Statements This press release contains “forward-looking statements” within the meaning of the U.S. federal securities laws. Statements contained herein that are not clearly historical in nature are forward-looking, and the words “anticipate,” “believe,” “continues,” “expect,” “estimate,” “intend,” “project,” “plan,” “is designed to,” “potential,” “predict,” “objective” and similar expressions and future or conditional verbs such as “will,” “would,” “should,” “could,” “might,” “can,” and “may,” or the negative of these are generally intended to identify forward-looking statements. Such forward-looking statements involve known and unknown risks, uncertainties, and other important factors that may cause CENTOGENE’s actual results, performance, or achievements to be materially different from any future results, performance, or achievements expressed or implied by the forward- looking statements. Such risks and uncertainties include, among others, our ability to achieve the revenue levels set forth in our guidance, negative economic and geopolitical conditions and instability and volatility in the worldwide financial markets, possible changes in current and proposed legislation, regulations and governmental policies, pressures from increasing competition and consolidation in our industry, the expense and uncertainty of regulatory approval, including from the U.S. Food and Drug Administration, our reliance on third parties and collaboration partners, including our ability to manage growth, our ability to execute our business plan and strategy, including to enter into new client relationships, our ability to execute on our announced strategic alternatives process, our dependency on the rare disease industry, our ability to manage international expansion, our reliance on key personnel, our reliance on intellectual property protection, fluctuations of our operating results due to the effect of exchange rates, our ability to streamline cash usage, our continued ongoing compliance with covenants linked to financial instruments, including timing requirements with respect to our strategic alternatives process, our requirement for additional financing, and our ability to continue as a going concern, or other factors. For further information on the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to CENTOGENE’s business in general, see CENTOGENE’s risk factors set forth in CENTOGENE’s Form 20-F filed on May 15, 2024, with the Securities and Exchange Commission (the “SEC”) and subsequent filings with the SEC. Any forward-looking statements contained in this press release speak only as of the date hereof, and CENTOGENE specifically disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events, or otherwise. Non-IFRS Measures This document may contain summarized, non-audited, or non-GAAP financial information. The information contained herein should therefore be considered as a whole and in conjunction with all the public information regarding the Company available, including any other documents released by the Company that may contain more detailed information. Adjusted EBITDA, adjusted EBITDA as a percentage of sales, working capital as a percentage of sales, adjusted EBITDA margin, working capital, adjusted net profit, adjusted profit per share, adjusted gross debt, and net cash/debt are non-IFRS financial metrics that management uses in its decision making. CENTOGENE has included these financial metrics to provide supplemental measures of its performance. The Company believes these metrics are important and useful to investors because they eliminate items that have less bearing on the Company’s current and future operating performance and highlight trends in its core business that may not otherwise be apparent when relying solely on IFRS financial measures. CONTACT CENTOGENE Melissa Hall Corporate Communications Press@centogene.com Lennart Streibel Investor Relations IR@centogene.com Centogene N.V. Consolidated statements of comprehensive loss for the years ended December 31, 2023, 2022 and 2021 (in EUR k) Note 2023 2022 2021 Revenue 7.2 48,536 47,473 42,234 Cost of sales 31,287 27,712 28,735 Gross profit 17,249 19,761 13,499 Research and development expenses 12,361 17,488 19,297 General administrative expenses 32,588 32,587 43,480 Selling expenses 12,564 9,924 9,326 Impairment of financial assets 812 — 827 Gain on reversal of financial asset impairment — 432 — Other operating income 8.1 11,848 3,774 2,894 Other operating expenses 8.2 431 741 86 Operating loss (29,659 ) (36,773 ) (56,623 ) Losses from investments accounted for by the Equity method 15 (302 ) — — Gains/(losses) on changes in fair value of warrants 8.3 (159 ) 2,574 — Interest and similar income 3,293 512 3 Interest and similar expenses 8,418 4,909 802 Financial costs, net 8.3 (5,284 ) (1,823 ) (799 ) Loss before taxes from continuing operations (35,245 ) (38,596 ) (57,422 ) Income taxes expenses 10 287 107 (70 ) Loss for the year from continuing operations (35,532 ) (38,703 ) (57,352 ) Net income from discontinued operations, net of tax 9 — 6,862 11,106 Loss for the period (35,532 ) (31,841 ) (46,246 ) Other comprehensive income/(loss), all attributable to equity holders of the parent (271 ) (76 ) 543 Total comprehensive loss (35,803 ) (31,917 ) (45,703 ) Attributable to: — — Equity holders of the parent (35,803 ) (31,917 ) (45,801 ) Non‑controlling interests from continuing operations 25 — — 98 Non‑controlling interests from discontinued operations — — — (35,803 ) (31,917 ) (45,703 ) Net loss per share - Basic and diluted from (in EUR) Continuing operations 11 (1.27 ) (1.45 ) (2.53 ) Loss attributable to parent 11 (1.27 ) (1.19 ) (2.04 ) The accompanying notes form an integral part of these consolidated financial statements Centogene N.V. Consolidated statements of financial position as of December 31, 2023 and 2022 (in EUR k) Assets Note Dec 31, 2023 Dec 31, 2022 Non‑current assets Intangible assets 12 6,850 7,400 Property, plant and equipment 13 5,643 6,808 Right-of-use assets 14 13,635 15,351 Investment in Joint Venture 15 2,784 — Derivative assets 23.1 799 510 Other assets 17 3,425 2,911 33,136 32,980 Current assets Inventories 16 2,463 1,819 Trade receivables and contract assets 17 19,415 16,548 Other assets 17 3,042 5,514 Cash and cash equivalents 18 19,099 35,951 44,019 59,832 77,155 92,812 Equity and liabilities Note Dec 31, 2023 Dec 31, 2022 Equity Issued capital 19 3,478 3,307 Capital reserve 19 148,308 145,369 Accumulated deficit and other reserves (177,068 ) (141,265 ) Non‑controlling interests — — (25,282 ) 7,411 Non‑current liabilities Non‑current loans 21.1 39,880 40,051 Lease liabilities 21.1 12,399 13,125 Deferred tax liabilities 10 407 35 Government grants 21.2 5,701 6,687 Derivative liabilities 21.2, 23 242 376 Warrant liability 21.2, 23 394 260 Other liabilities 21.2, 22 48 202 59,071 60,736 Current liabilities Government grants 21.2 984 1,263 Current loans 21.1 25,882 4,635 Lease liabilities 21.1 2,178 2,311 Liabilities from income taxes 87 89 Trade payables 21.2 5,628 6,317 Other liabilities 21.2, 22 8,607 10,050 43,366 24,665 77,155 92,812 The accompanying notes form an integral part of these consolidated financial statements Centogene N.V. Consolidated statements of cash flows for the years ended December 31, 2023, 2022 and 2021 (in EUR k) Note 2023 2022 2021 Operating activities Loss before taxes from continuing operations (35,245 ) (38,596 ) (57,422 ) Income before taxes from discontinued operations 9 — 6,875 11,152 Loss before taxes (35,245 ) (31,721 ) (46,270 ) Adjustments to reconcile earnings to cash flow from operating activities Amortization (including impairments) and depreciation 12,13,14 7,610 10,378 19,974 Government grant depreciation (1,265 ) — — Inventory write-off 16 226 — 1,795 Interest income 8.3 (209 ) — (3 ) Interest expense 8.3 8,201 4,909 851 Gain on the disposal of property, plant and equipment (56 ) (754 ) (18 ) Gain on the sales of IP to Joint Venture 8.1 (7,549 ) — — Expected credit loss allowances on trade receivables and contract assets 23.2 761 — 827 Share‑based payment expenses 22 2,929 (16 ) 8,035 Tax Expense — (89 ) — Profit or loss from financial instruments FV adjustments 8.3 (263 ) (2,574 ) — Loss from Joint Venture equity method 15 302 — — Other non‑cash items (34 ) (1,430 ) (821 ) Net foreign exchange differences (2,438 ) 963 — Interest received 209 — — Changes in operating assets and liabilities: Inventories 16 (870 ) 2,050 5,741 Trade receivables and contract assets 17 (3,628 ) 6,914 4,855 Other assets 17 1,858 - 1,828 Trade payables 21.2 (689 ) (4,935 ) (20,484 ) Other liabilities (1,599 ) (10,182 ) 1,952 Thereof cash flow (used in) continuing operating activities (31,749 ) (35,497 ) (42,635 ) Thereof cash flow from discontinued operating activities — 9,009 20,897 Cash flow (used in)/from operating activities (31,749 ) (26,488 ) (21,739 ) Investing activities Cash paid for investments in intangible assets 12 (2,239 ) (1,727 ) (2,787 ) Cash paid for investments in property, plant and equipment 13 (40 ) (367 ) (2,915 ) Cash paid for investment in Joint Venture 15 (4,973 ) — — Grants received for investment in property, plant and equipment 21.2 — 506 168 Cash received from the disposals of property, plant and equipment 93 855 171 Cash received from sale of IP to Joint Venture 15 9,436 — — Interest received — — 3 Thereof cash flow from/(used in) in continuing investing activities 2,277 (1,553 ) (2,494 ) Thereof cash flow from/(used in) discontinued investing activities — 820 (2,866 ) Cash flow used in investing activities 2,277 (733 ) (5,360 ) Financing activities Cash received from the issuance of shares 19 — 12,140 — Cash received from issuance of warrants — 2,833 — Cash paid for acquisition of non-wholly owned subsidiary — (1 ) — Cash received from loans 21, 23.2 25,500 40,568 1,772 Cash repayments of loans 21, 23.2 (3,374 ) — (464 ) Cash repayments of lease liabilities 21, 23.2 (3,095 ) (4,314 ) (4,244 ) Interest paid 8.3 (5,987 ) (4,909 ) (267 ) Thereof net cash flow from/(used in) continuing financing activities 13,044 46,798 (2,403 ) Thereof net cash flow used in discontinued financing activities — (481 ) (800 ) Cash flow from financing activities 13,044 46,317 (3,203 ) Changes in cash and cash equivalents (16,428 ) 19,096 (30,302 ) Cash and cash equivalents at the beginning of the period 35,951 17,818 48,156 Effect of movements in exchange rates on cash held (424 ) (963 ) (36 ) Cash and cash equivalents at the end of the period 19,099 35,951 17,818 The accompanying notes form an integral part of these consolidated financial statements

An undergraduate student turns her honor's thesis into a peer-reviewed publication on schizophrenia research. As an undergraduate student in The University of Texas at Arlington's Honors College, Hannah Selvarathinam knew she wanted to conduct research. Near the end of her first year at UTA, the Keller native reached out to the lab of biology Assistant Professor Piya Ghose. "Hannah has been a very impressive scholar from Day 1," Ghose said. "She had the foresight to reach out for research opportunities very early on." Ghose brought Selvarathinam in to work on one of the lab's core projects related to the genetics of cell death, modeled in the roundworm Caenorhabditis elegans. Selvarathinam's work eventually led to her honors thesis, focused on brain health and behavior. The result is a new peer-reviewed publication detailing the genes affecting worm behavior that also are relevant to neurological disease in humans. "Through combing the literature and discussing her findings with me, she was able to make connections between the genes she was already studying as part of her cell biology project with her undergraduate psychology major and interests in human health," Ghose said. "Hannah drove this project and regularly communicated with me for guidance and to share her exciting results. She took on the brave task of adapting and optimizing a published behavioral protocol for her project, which is exceptional for such a young trainee." For her study, Selvarathinam leveraged the fact that worms have similarities in their genes to humans and predictable behaviors that are easy to study. She proceeded to optimize an experi-mental protocol that aimed to link the mental illness schizophrenia to neurodegenerative disease using worm behavior. "Normally, worms spend much of their time eating. But if they are interrupted by physical insult, they briefly stop in a manner similar to the startle response in humans," Selvarathinam said. Schizophrenia has similar symptoms, and one hallmark is abnormal reactivity to a continued stimulus. Essentially, individuals with the illness take longer to become accustomed to a stimulus than those without. "What we found in our study is that healthy worms, as expected, momentarily stopped eating when they were exposed to a stimulus, but soon continued to eat again," Selvarathinam explained. "But for worms with mutations in many of the neurodegeneration genes, such as with those related to hereditary spastic paraplegia and Alzheimer's disease, they continued to eat even after being exposed to the stimulus, which shows a heightened startle response. "Our publication adds another building block to our understanding of brain disease in the hopes of finding a cure and also highlights C.elegans as a powerful model organism to pursue this goal." After completing her honors thesis and graduating in winter 2022, Selvarathinam began working as a technician in Ghose's lab while preparing applications for medical school. She's now on track with another peer-reviewed publication for work she has done assisting on one of the lab's cell death projects. "My research experience complements my goal to practice medicine by teaching me many transferable skills, such as reading scientific literature and applying my knowledge to solve problems and answer interesting questions," Selvarathinam said. "I am grateful for the opportunities I have had at UTA and for the chance to work with the Ghose Lab team. I am also thankful to the Honors College for its wonderful capstone project program. I encourage students to reach out to professors and seek out research opportunities here at UTA."

Company will perform plasmid DNA production in support of early phase trials for SPG56 Hereditary Spastic Paraplegia WILMINGTON, Mass.--(BUSINESS WIRE)-- Charles River Laboratories International, Inc. (NYSE: CRL) and Genetic Cures for Kids Inc (GC4K), an Australian non-profit foundation focused on research programs and clinical trials to find cures for rare diseases, today announced a plasmid DNA contract development and manufacturing organization (CDMO) collaboration. Founded in 2021 by Golden and Chris Whitrod, parents of now 4 year old Tallulah Moon, GC4K’s first mission, known as Our Moon’s Mission, is to develop a gene therapy for the treatment of Hereditary Spastic Paraplegia Type 56 (SPG56). SPG56 is a debilitating neurodegenerative disease that robs children of the ability to walk, talk, and move independently. What is SPG56? SPG56 manifests as a progressive neurological disease characterised by varying degrees of spasticity and muscle weakness. This degenerative disease typically begins with motor and cognitive regression in childhood and continuously worsens through life. Additional clinical manifestations include intellectual disability, dystonia, cerebellar ataxia, subclinical peripheral neuropathy, seizures, and visual impairment. Tragically, there is currently no treatment for this ultra-rare genetic disease. In addition, limited access to genetic testing means diagnoses are hard to achieve and as such, fewer than one in 1 million people have ever been diagnosed with SPG56 globally. GC4K is leading a gene therapy program that aim not to only cure SPG56, but to create a replicable framework that paves the way to develop treatments for some of the 7,000 other genetic diseases in the world. Plasmid DNA Manufacturing Services GC4K will leverage Charles River’s established plasmid platform, eXpDNA™, and premier expertise in plasmid DNA production, including High Quality (HQ) plasmid which combines key features of good manufacturing practice (GMP) manufacture with a rapid turnaround to accelerate time to clinic. As a critical starting material in the development of cell and gene therapies, in 2022 Charles River opened a state-of-the-art HQ Plasmid Manufacturing Center of Excellence dedicated to addressing global supply shortages and support the growing needs of customers. In recent years, Charles River has significantly broadened its cell and gene therapy portfolio to simplify complex supply chains and meet growing demand for plasmid DNA, viral vector, and cell therapy services. Combined with the Company’s legacy testing capabilities, Charles River offers a comprehensive “concept-to-cure” advanced therapies solution. To learn more about plasmid DNA production, watch Advancements in Manufacturing for Rapid Delivery of pDNA Starting Materials, presented by Andrew Frazer, PhD, Associate Director, Scientific Solutions, Gene Therapy CDMO Services, on-demand: Approved Quotes “The opportunity to work with GC4K is exactly why we do what we do, playing a role in delivering potentially curative treatments to patients suffering with an ultra-rare disease. We are excited to support the team as they work through the next stage of their program development.” - Kerstin Dolph, Corporate Senior Vice President, Biologics Solutions, Charles River “What was once a tragic diagnosis, has the potential to become a triumph, as we draw nearer to finding a cure for SPG56. With the advancement of this groundbreaking treatment, our partnership with Charles River is bringing us closer to aiding children like Tallulah, who, until now, have faced the daily hardships of SPG56 without any glimmer of hope. We're truly excited to have the backing of this skilled team as we gear up to produce this historic treatment. By making this announcement on Giving Tuesday, we are giving thanks to Charles River. This symbolic occasion not only commends the generosity of CRL in supporting GC4K’s quest to cure SPG56, but also to recognizes the broader goal that our partnership is striving for - a brighter future for all children living with rare genetic diseases." - Golden Whitrod, Tallulah Moon’s mother and Co-Founder and President, Genetic Cures for Kids, Inc. About Charles River Charles River provides essential products and services to help pharmaceutical and biotechnology companies, government agencies and leading academic institutions around the globe accelerate their research and drug development efforts. Our dedicated employees are focused on providing clients with exactly what they need to improve and expedite the discovery, early-stage development and safe manufacture of new therapies for the patients who need them. To learn more about our unique portfolio and breadth of services, visit . Genetic Cures for Kids, Inc., and Our Moon’s Mission GC4K is a family-driven research foundation at the forefront of genetic research, with a primary objective to not only develop a cure for SPG56 but also to establish a reproducible framework that can catalyze the development of treatments for some of the 7,000 known genetic diseases worldwide. The foundation's campaign to cure SPG56 is "Our Moon's Mission", named in honor of the founders’ daughter Tallulah Moon, who lives with SPG56. By leveraging crowdfunding to assemble an expert international research team, which has since gained recognition and funding from the Australian Government and is guided by an Independent Scientific Advisory Board, GC4K aims to expedite the development of effective SPG56 therapies for clinical use. Additionally, the foundation is dedicated to advocating for 'rare families' and increasing awareness of rare diseases. To learn more about GC4K's commitment to advancing genetic research and to support the foundation's mission to cure SPG56, please visit